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Article

Next steps for the optimization of exon therapy for Duchenne muscular dystrophy

19 Jan 2023

Abstract excerpt

INTRODUCTION: It is established that the exon-skipping approach can restore dystrophin in Duchenne muscular dystrophy (DMD) patients. However, dystrophin restoration levels are low, and the field is evolving to provide solutions for improved exon skipping. DMD is a neuromuscular disorder associated with chronic muscle tissue loss attributed to the lack of dystrophin, which causes muscle inflammation, fibrosis...

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PMID: 36655939
DOI: 10.1080/14712598.2023.2169070

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